A decisive first step was taken this week in the Vyndaqel market access procedure for TTR amyloid cardiomyopathies.
This Thursday 12 December, the Committee for Human Use for Medicinal Products (CHMP) of the European Union adopted a favorable opinion recommending the approval of VYNDAQEL (tafamidis), an oral capsule at 61 mg once a day, for the treatment of wild-type or hereditary amyloidosis based on transthyretin in adult patients with cardiomyopathy (ATTR-CM).
The European Medicines Agency has published this opinion: https://www.ema.europa.eu/en/medicines/human/summaries-opinion/vyndaqel
This opinion will soon be confirmed by a European Commission decision which will open the door to approval procedures in the different countries.
A press release from Pfizer commented on this publication: https://www.businesswire.com/news/home/20191213005202/en/Pfizer-Receives-Positive-CHMP-Opinion-VYNDAQEL%C2%AE-Patients
"If approved by the European Commission (EC), VYNDAQEL® will be the first pharmacologic therapy in the EU for patients with transthyretin amyloid cardiomyopathy-"
The International Alliance of Patient Associations in the same press release welcomed this announcement:
"For those living with ATTR-CM, a progressive and fatal rare disease, there are currently no available pharmacologic treatments for patients," said Jean-Christophe Fidalgo, President of the Amyloidosis Alliance. "The Amyloidosis Alliance applauds the CHMP opinion, and we hope the EC will swiftly approve VYNDAQEL for ATTR-CM so patients can receive timely access to this medicine."